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FDA Approves First Gene Therapy Treatment for Duchenne Muscular Dystrophy Developed at the Abigail Wexner Research Institute at Nationwide Children’s Hospital

Jerry Mendell, MD

The Abigail Wexner Research Institute at Nationwide Children’s Hospital praised the Food and Drug Administration (FDA) for its accelerated approval of SRP-9001/ELEVIDYS for Duchenne muscular dystrophy (DMD) following decades of research in its Center for Gene Therapy to help patients with neuromuscular diseases.

The FDA approved the treatment for pediatric patients 4-5 years old with DMD. Jerry Mendell, MD, principal investigator in the Center for Gene Therapy at the Abigail Wexner Research Institute, led the first Phase I study of a systemic gene therapy for DMD. Dr. Mendell saw his first patient with DMD more than 50 years ago, igniting his passion to develop new treatments for patients with neuromuscular diseases.

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